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Study of Regorafenib with Chemotherapy for Newly Diagnosed Patients with Metastatic Ewing Sarcoma

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a type of cancer called Ewing sarcoma, which is a rare cancer that affects bones or the soft tissue around bones. The study is specifically for patients who have been newly diagnosed with Ewing sarcoma that has spread to other parts of the body, except for the lungs or the lining around the lungs. The treatment being tested in this study is a combination of a drug called regorafenib and standard chemotherapy. Regorafenib is a medication that is taken by mouth and is being tested in two forms: as granules and as film-coated tablets. The purpose of the study is to find the best dose of regorafenib when used with chemotherapy for treating this type of cancer.

Participants in the study will receive the combination of regorafenib and chemotherapy. The study will monitor how the body responds to the treatment and any side effects that may occur. The study will also look at how the cancer responds to the treatment, including whether it shrinks or stops growing. The study will last for several years, and participants will be closely monitored throughout the process to ensure their safety and to gather important information about the treatment's effectiveness.

The study aims to improve the treatment options for patients with Ewing sarcoma by determining the most effective way to use regorafenib in combination with chemotherapy. This research could potentially lead to better outcomes for patients with this challenging form of cancer. Participants will be contributing to important research that may help future patients with Ewing sarcoma.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, you will be required to provide written informed consent. This is a document that confirms your understanding of the study and your agreement to participate.

    You will need to confirm that you can swallow and retain oral medication, as the treatment involves taking medication by mouth.

  2. Step 2

    Initial assessments

    Before starting the treatment, several health assessments will be conducted. These include blood tests to check your blood cell counts, liver function, kidney function, and heart function.

    A pregnancy test will be conducted for female patients of childbearing potential.

    Your blood pressure will be measured to ensure it is adequately controlled.

  3. Step 3

    Treatment cycle 1

    The treatment involves taking a medication called regorafenib in combination with standard chemotherapy. Regorafenib is taken orally in the form of granules or film-coated tablets.

    The first cycle of treatment lasts for 28 days. During this time, you will be monitored for any side effects or dose-limiting toxicities, which are side effects that may require adjusting the dose of the medication.

  4. Step 4

    Monitoring and adjustments

    Throughout the treatment, regular monitoring will be conducted to assess your response to the medication and to check for any adverse effects.

    If any severe side effects occur, the dosage of regorafenib or chemotherapy may be adjusted, or the treatment may be paused or stopped.

  5. Step 5

    Subsequent treatment cycles

    After the first cycle, additional cycles of treatment will continue, with each cycle lasting 28 days.

    The treatment will continue until the study's end date or until it is determined that the treatment is no longer beneficial or safe for you.

  6. Step 6

    End of study

    The study is estimated to end by June 30, 2026. At the end of the study, a final assessment will be conducted to evaluate your overall health and response to the treatment.

    You will be informed about the results of the study and any further steps that may be necessary for your health care.

Who can join the trial?

16 criteria

  • Must have a newly diagnosed condition called metastatic Ewing sarcoma, which means the cancer has spread to other parts of the body, but not just to the lungs or the lining around the lungs.
  • The type of cancer must be confirmed through specific tests, either as Ewing sarcoma of the bone or soft tissue, or a similar type that does not have a specific gene change called EWSR1 gene rearrangement.
  • Must be able to swallow and keep down oral medication.
  • Must provide written consent to participate, which can be given by the patient or their parent/legal guardian.
  • Must have metastatic disease, meaning the cancer has spread to other parts of the body.
  • Must be between the ages of 2 years and less than 50 years old.
  • Must be medically fit to receive standard treatment for Ewing sarcoma and a drug called regorafenib. This includes having:
    • An Absolute Neutrophil Count (ANC) of at least 0.75x109/L and platelets of at least 75x109/L.
    • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels no more than 5 times the upper limit of normal (ULN).
    • Bilirubin levels no more than 2 times the ULN.
    • Creatinine levels less than 2 times the ULN or a creatinine clearance greater than 60 ml/min/1.73 m2.
    • International normalized ratio (INR) and Partial thromboplastin time (PTT) no more than 1.5 times the ULN.
    • Must have adequate heart function, shown by a left ventricular ejection fraction (LVEF) of at least 50% as measured by an ultrasound of the heart.
    • Must have controlled blood pressure, with or without medication. For those under 18, blood pressure should be below the 95th percentile for their sex, age, and height. For those over 18, blood pressure should be 150/90 mm Hg or lower.
    • Must not have received any prior treatment for Ewing sarcoma, except for surgery.
    • Female patients who can have children must have a negative pregnancy test within 7 days before joining the study.
    • Must agree to use highly effective birth control during the study and for 12 months after the last treatment for females, or 6 months for males, if applicable.

Who cannot join the trial?

1 criterion

  • Patients who have been diagnosed with Ewing sarcoma that has spread to areas other than the lungs or the lining around the lungs (pleura) cannot participate.
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Investigated drugs

  • Regorafenib

    is a medication used in this trial to help treat Ewing sarcoma, a type of cancer. It works by blocking certain proteins that cancer cells need to grow and spread. In this study, regorafenib is being tested in combination with other chemotherapy drugs to see if it can improve treatment outcomes for patients with this type of cancer.

  • VDC/IE

    is a combination of chemotherapy drugs used as a standard treatment for Ewing sarcoma. VDC stands for vincristine, doxorubicin, and cyclophosphamide, while IE stands for ifosfamide and etoposide. These drugs work together to kill cancer cells or stop them from growing. In this trial, VDC/IE is used alongside regorafenib to see if the combination is more effective in treating patients with newly diagnosed metastatic Ewing sarcoma.

What is already known about the treatment

Regorafenib – Regorafenib is administered orally in the form of tablets and is currently being studied in combination with conventional chemotherapy for treating newly diagnosed patients with metastatic Ewing sarcoma, excluding those with only lung or pleura metastases. It is in the clinical trial phase to determine the optimal dose when used alongside standard chemotherapy. The main therapeutic indication for regorafenib in this trial is to treat Ewing sarcoma, a type of cancer that occurs in bones or soft tissue. At the molecular level, regorafenib works by blocking certain proteins that promote cancer cell growth, effectively slowing down or stopping the progression of the disease. It is classified pharmacologically as a multikinase inhibitor, which means it targets multiple enzymes involved in cancer cell proliferation.

Investigated diseases

Ewing Sarcoma – Ewing sarcoma is a rare type of cancer that occurs in bones or in the soft tissue around the bones. It primarily affects children and young adults. The disease often begins in the long bones of the arms and legs, pelvis, or chest. It can spread to other parts of the body, including bone marrow and other organs. The progression of Ewing sarcoma involves the growth of a tumor, which can cause pain and swelling in the affected area. As the disease advances, it may lead to additional symptoms such as fever, fatigue, and weight loss.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2023-503322-39-00Protocol codeCSET2022/3545Estimated enrolment24 patientsSponsorInstitut Gustave Roussy

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).